In short
Clinical trials of Sodium Valproate are studying whether it helps in several diseases, including epilepsy, cancer, Wolfram syndrome, tuberous sclerosis complex, bipolar depression, and adenomyosis. These studies look at safety, symptom control, recovery, and other outcomes in different patient groups such as children, adults, and critically ill patients.
Key points
- Sodium Valproate is being studied in a wide range of clinical trials, from early safety research to later studies of benefit. The trials include people with epilepsy-related emergencies, cancer, Wolfram syndrome, tuberous sclerosis complex, bipolar depression, adenomyosis, and other conditions. Some studies compare Sodium Valproate with other treatments, while others test it as part of a combination plan. The main outcomes include symptom improvement, functional recovery, hearing preservation, pain reduction, neuropsychologic development, and progression-free survival. The trial data also show different age groups and patient types, including children, adults, and critically ill patients. Overall, the research is focused on whether Sodium Valproate can improve disease-specific outcomes in the studied populations.
Clinical trials overview
The trial data show that Sodium Valproate is being studied in several different diseases, not just one condition.
These studies include interventional trials, which means the researchers give a treatment and then measure what happens.
The trial phases range from Phase 1 to Phase 4, so the research includes early safety work and later studies that look at treatment benefit in larger groups.
Neurology and emergency seizure studies
Several trials focus on seizure-related conditions, including status epilepticus, which is a medical emergency involving ongoing or repeated seizures.
In NCT05263674, the FAST TRIAL, researchers compare intravenous Sodium Valproate with other anti-seizure and sedation treatments in people with status epilepticus, and the main outcome is treatment failure after 24 hours.
In NCT06549426, the TELSTAR-2 trial studies comatose cardiac arrest patients with status epilepticus on continuous EEG, and it asks whether stepwise anti-seizure treatment improves functional recovery at six months.
The TELSTAR-2 primary outcome is the score on the extended Glasgow Outcome Scale (eGOS), which measures recovery after brain injury or severe illness.
Cancer studies
One Phase 1 study, NCT06199557, looks at Sodium Valproate in patients with acute myeloid leukemia or high-risk myelodysplastic syndrome who are considered unfit for standard chemotherapy.
This trial tests combinations with hydroxyurea or 6-mercaptopurine and focuses on safety and tolerability, including adverse events, dose-limiting toxicities, lab tests, and physical exams.
It also measures early signs of clinical benefit and changes in Eastern Cooperative Oncology Group (ECOG) performance status, which is a score that shows how well a person can carry out daily activities.
In 2024-516844-25-01, the REVOLUTION study tests whether adding valproic acid and Sodium Valproate to bevacizumab and oxaliplatin/fluoropyrimidine regimens can improve progression-free survival in people with RAS-mutated metastatic colorectal cancer.
Pediatric and rare disease studies
The AUDIOWOLF study, NCT04940572, is a Phase 2 trial in people with Wolfram syndrome due to a monogenic mutation.
Its main goal is to see whether daily Sodium Valproate can preserve hearing over three years, using audiometry and high-frequency hearing tests.
Another Phase 2 study, 2022-502332-39-00, includes children with tuberous sclerosis complex under 4 months of age and measures neuropsychologic outcome at 24 months using the cognitive scale of the Bayley Scales of Infant and Toddler Development III (BSID-III).
In the ependymoma program NCT02265770, Sodium Valproate appears in one treatment stratum for children unable to receive radiation therapy, where the study evaluates progression-free survival when valproate is added to the main chemotherapy strategy.
Psychiatry studies
Two Phase 3 studies include people with mood and other psychiatric disorders.
NCT05973786 studies a six-week intensified drug plan for bipolar depression in people who had a first-time treatment failure on first-line treatment, and it measures change in symptom severity using the Montgomery-Åsberg Depression Rating Scale (MADRS).
NCT05603104 was a larger Phase 3 study in schizophrenia, schizoaffective disorder, schizophreniform disorder, major depressive disorder, and bipolar depression, but the trial status is withdrawn.
That withdrawn study also measured symptom change, using the Positive and Negative Syndrome Scale for schizophrenia and MADRS for major depressive disorder and bipolar depression.
Women's health study
In 2025-523076-23-00, researchers are studying women of reproductive age with adenomyosis in a Phase 2 trial.
The trial compares a plan that includes a single intralesional administration of valproic acid followed by oral dosing with placebo-based comparators, and it looks at pelvic pain and rescue medication use over 29 days.
The primary endpoint uses change in AAPP, which is the trial's pain measure, and it also records how much ibuprofen is needed as rescue medicine.
Main outcomes used in the trials
The studies use different endpoints because they are asking different clinical questions.
- Functional recovery is used in the post-cardiac arrest trial and is measured by eGOS at six months.
- Safety and tolerability are central in the leukemia trial, where the team tracks adverse events, exams, and lab results.
- Hearing preservation is the key outcome in Wolfram syndrome, with testing over three years.
- Progression-free survival is used in the colorectal cancer and ependymoma studies to see how long the disease stays controlled.
- Symptom severity is measured in the bipolar depression study using MADRS, while the withdrawn psychiatric study used both MADRS and PANSS depending on the diagnosis.
- Neuropsychologic outcome is measured in young children with tuberous sclerosis complex using blinded testing at 24 months.